Category Archives: Genetic Engineering

Our society has evolved so much, can we still say that we are part of Nature? If not, should we worry and what should we do about it? Poppy, 21, Warwick.

Such is the extent of our dominion on Earth, that the answer to questions around whether we are still part of nature and whether we even need some of it rely on an understanding of what we want as Homo sapiens. And to know what we want, we need to grasp what we are.

It is a huge question but they are the best. And as a biologist, here is my humble suggestion to address it, and a personal conclusion. You may have a different one, but what matters is that we reflect on it.

Perhaps the best place to start is to consider what makes us human in the first place, which is not as obvious as it may seem.

This article is part of Lifes Big QuestionsThe Conversations new series, co-published with BBC Future, seeks to answer our readers nagging questions about life, love, death and the universe. We work with professional researchers who have dedicated their lives to uncovering new perspectives on the questions that shape our lives.

Many years ago, a novel written by Vercors called Les Animaux dnaturs (Denatured Animals) told the story of a group of primitive hominids, the Tropis, found in an unexplored jungle in New Guinea, who seem to constitute a missing link.

However, the prospect that this fictional group may be used as slave labour by an entrepreneurial businessman named Vancruysen forces society to decide whether the Tropis are simply sophisticated animals or whether they should be given human rights. And herein lies the difficulty.

Human status had hitherto seemed so obvious that the book describes how it is soon discovered that there is no definition of what a human actually is. Certainly, the string of experts consulted anthropologists, primatologists, psychologists, lawyers and clergymen could not agree. Perhaps prophetically, it is a layperson who suggested a possible way forward.

She asked whether some of the hominids habits could be described as the early signs of a spiritual or religious mind. In short, were there signs that, like us, the Tropis were no longer at one with nature, but had separated from it, and were now looking at it from the outside with some fear.

It is a telling perspective. Our status as altered or denatured animals creatures who have arguably separated from the natural world is perhaps both the source of our humanity and the cause of many of our troubles. In the words of the books author:

All mans troubles arise from the fact that we do not know what we are and do not agree on what we want to be.

We will probably never know the timing of our gradual separation from nature although cave paintings perhaps contain some clues. But a key recent event in our relationship with the world around us is as well documented as it was abrupt. It happened on a sunny Monday morning, at 8.15am precisely.

The atomic bomb that rocked Hiroshima on August 6 1945, was a wake-up call so loud that it still resonates in our consciousness many decades later.

The day the sun rose twice was not only a forceful demonstration of the new era that we had entered, it was a reminder of how paradoxically primitive we remained: differential calculus, advanced electronics and almost godlike insights into the laws of the universe helped build, well a very big stick. Modern Homo sapiens seemingly had developed the powers of gods, while keeping the psyche of a stereotypical Stone Age killer.

We were no longer fearful of nature, but of what we would do to it, and ourselves. In short, we still did not know where we came from, but began panicking about where we were going.

We now know a lot more about our origins but we remain unsure about what we want to be in the future or, increasingly, as the climate crisis accelerates, whether we even have one.

Arguably, the greater choices granted by our technological advances make it even more difficult to decide which of the many paths to take. This is the cost of freedom.

I am not arguing against our dominion over nature nor, even as a biologist, do I feel a need to preserve the status quo. Big changes are part of our evolution. After all, oxygen was first a poison which threatened the very existence of early life, yet it is now the fuel vital to our existence.

Similarly, we may have to accept that what we do, even our unprecedented dominion, is a natural consequence of what we have evolved into, and by a process nothing less natural than natural selection itself. If artificial birth control is unnatural, so is reduced infant mortality.

I am also not convinced by the argument against genetic engineering on the basis that it is unnatural. By artificially selecting specific strains of wheat or dogs, we had been tinkering more or less blindly with genomes for centuries before the genetic revolution. Even our choice of romantic partner is a form of genetic engineering. Sex is natures way of producing new genetic combinations quickly.

Even nature, it seems, can be impatient with itself.

Advances in genomics, however, have opened the door to another key turning point. Perhaps we can avoid blowing up the world, and instead change it and ourselves slowly, perhaps beyond recognition.

The development of genetically modified crops in the 1980s quickly moved from early aspirations to improve the taste of food to a more efficient way of destroying undesirable weeds or pests.

In what some saw as the genetic equivalent of the atomic bomb, our early forays into a new technology became once again largely about killing, coupled with worries about contamination. Not that everything was rosy before that. Artificial selection, intensive farming and our exploding population growth were long destroying species quicker than we could record them.

The increasing silent springs of the 1950s and 60s caused by the destruction of farmland birds and, consequently, their song was only the tip of a deeper and more sinister iceberg. There is, in principle, nothing unnatural about extinction, which has been a recurring pattern (of sometimes massive proportions) in the evolution of our planet long before we came on the scene. But is it really what we want?

The arguments for maintaining biodiversity are usually based on survival, economics or ethics. In addition to preserving obvious key environments essential to our ecosystem and global survival, the economic argument highlights the possibility that a hitherto insignificant lichen, bacteria or reptile might hold the key to the cure of a future disease. We simply cannot afford to destroy what we do not know.

But attaching an economic value to life makes it subject to the fluctuation of markets. It is reasonable to expect that, in time, most biological solutions will be able to be synthesised, and as the market worth of many lifeforms falls, we need to scrutinise the significance of the ethical argument. Do we need nature because of its inherent value?

Perhaps the answer may come from peering over the horizon. It is somewhat of an irony that as the third millennium coincided with decrypting the human genome, perhaps the start of the fourth may be about whether it has become redundant.

Just as genetic modification may one day lead to the end of Homo sapiens naturalis (that is, humans untouched by genetic engineering), we may one day wave goodbye to the last specimen of Homo sapiens genetica. That is the last fully genetically based human living in a world increasingly less burdened by our biological form minds in a machine.

If the essence of a human, including our memories, desires and values, is somehow reflected in the pattern of the delicate neuronal connections of our brain (and why should it not?) our minds may also one day be changeable like never before.

And this brings us to the essential question that surely we must ask ourselves now: if, or rather when, we have the power to change anything, what would we not change?

After all, we may be able to transform ourselves into more rational, more efficient and stronger individuals. We may venture out further, have greater dominion over greater areas of space, and inject enough insight to bridge the gap between the issues brought about by our cultural evolution and the abilities of a brain evolved to deal with much simpler problems. We might even decide to move into a bodiless intelligence: in the end, even the pleasures of the body are located in the brain.

And then what? When the secrets of the universe are no longer hidden, what makes it worth being part of it? Where is the fun?

Gossip and sex, of course! some might say. And in effect, I would agree (although I might put it differently), as it conveys to me the fundamental need that we have to reach out and connect with others. I believe that the attributes that define our worth in this vast and changing universe are simple: empathy and love. Not power or technology, which occupy so many of our thoughts but which are merely (almost boringly) related to the age of a civilisation.

Like many a traveller, Homo sapiens may need a goal. But from the strengths that come with attaining it, one realises that ones worth (whether as an individual or a species) ultimately lies elsewhere. So I believe that the extent of our ability for empathy and love will be the yardstick by which our civilisation is judged. It may well be an important benchmark by which we will judge other civilisations that we may encounter, or indeed be judged by them.

There is something of true wonder at the basis of it all. The fact that chemicals can arise from the austere confines of an ancient molecular soup, and through the cold laws of evolution, combine into organisms that care for other lifeforms (that is, other bags of chemicals) is the true miracle.

Some ancients believed that God made us in his image. Perhaps they were right in a sense, as empathy and love are truly godlike features, at least among the benevolent gods.

Cherish those traits and use them now, Poppy, as they hold the solution to our ethical dilemma. It is those very attributes that should compel us to improve the wellbeing of our fellow humans without lowering the condition of what surrounds us.

Anything less will pervert (our) nature.

To get all of lifes big answers, join the hundreds of thousands of people who value evidence-based news by subscribing to our newsletter. You can send us your big questions by email at bigquestions@theconversation.com and well try to get a researcher or expert on the case.

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Have humans evolved beyond nature and do we even need it? - The Conversation UK

A new type of cancer cell therapy could avoid some of the serious side effects commonly associated with CAR-T treatments, and possibly offer an easier path to developing "off-the-shelf" treatments, suggest findings from a small study led by researchers at the MD Anderson Cancer Center in Houston, Texas.

The results, which were published Wednesday in the New England Journal of Medicine, are from just 11 patients. Other factors, such as the use of postremission therapy, limit what conclusions can be drawn about the researchers' approach, which relies on "natural killer" cells rather than the T cells used in cellular drugs like Novartis' Kymriah.

Still, the data offer a glimpse into why Japanese drugmaker Takedaagreed last November to license the CAR NK cell therapy from MD Anderson, part of the company's broader push into cell and gene treatments. Some of the data published Wednesday was previously disclosed by the pharma.

The success of cancer immunotherapy, of which CAR-T treatments are a major part, has put T cells at the center of a now decade-long research revival in oncology.

But T cells are only one component of the body's immune system, and scientists in academia and in biotech are exploring whether other cellular defenders could be similarly recruited.

Researchers at MD Anderson have turned to natural killer cells, which by design recognize and attack cancers or other invaders. Such cells have been tested as an anti-cancer treatment before,but using genetic engineering to improve their tumor-killing properties, which the MD Anderson team has done, is a newer innovation.

"To my knowledge, this is the largest body of evidence on the use of CAR NK cells in patients with cancer," said Katayoun Rezvani, the study's corresponding author and a professor of stem cell transplantation and cellular therapy at MD Anderson, in an interview.

Using NK cells derived from cord blood, Rezvani and her colleagues engineered the cells to express a receptor for a protein called CD19, commonly found on the surface of B-cell malignancies like leukemia and lymphoma. They also added a gene for interleukin-15 to boost the expansion and persistence of the infused NK cells, which without engineering would typically disappear after about two weeks.

While the CAR-T treatments Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) also target CD19, they are made from a patient's own T cells, which are extracted and then engineered outside the body. The personalized process is time-consuming and laborious, hampering the commercial uptake of both Kymriahand Yescarta.

By using cord blood, Rezvani and her team are pursuing an allogeneic, or "off-the-shelf," approach to cell treatment something many consider to be the next step for the field.

Initial data look promising. Seven of the 11 treated patients, who had either chronic lymphocytic leukemia or non-Hodgkin lymphoma, responded to treatment, with the cancers of three going into remission.Most notably, none experienced cytokine release syndrome or neurotoxicity, two severe side effects that commonly occur in patients treated with CAR-T therapy.

"The lack of toxicity is very exciting here," wrote Stephan Grupp, an oncologist at Children's Hospital of Philadelphia and a leader in the CAR-T field, in comments emailed to BioPharma Dive. He was not involved with the MD Anderson study.

"We really think that this is something inherent to the biology of the natural killer cells, which means their profile of toxicity is different than that of T cells,"Rezvanisaid.

Study participants did have blood toxicities that researchers associated with the chemotherapy given prior to infusion of the CAR NK cells.

While positive, the results are limited by several factors which make drawing broader conclusions about the ultimate potential of the treatment difficult.

Five of the seven responding patients received postremission treatment, including stem cell transplants, Rituxan (rituximab) and Revlimid (lenalidomide), so researchers did not assess the duration of response to CAR NK therapy.

Additionally, a fresh CAR NK cell product was manufactured for each patient in this study, rather than using the cord blood to produce multiple therapies as would be envisioned for a true off-the-shelf product.

"I think the potential for this approach to be 'off-the-shelf' is also a little speculative at this time," wrote Grupp.

"We would need to see multiple patients treated from the same expanded product with no HLAmatching to know if 'off-the-shelf' is going to be part of the story here," he added, referring to the process by which patients are matched to donor cells.

If cord blood-derived CAR NK cells were able to be given without matching to a patient's HLA genotype, any resulting treatment could be used more widely. Nine patients were partially matched in the MD Anderson study, while the last two were treated without consideration of HLA type.

The MD Anderson researchers plan to continue enrolling patients in the study and are working with Takeda to design a larger, multi-center trial.

The drugmaker is planning to advance the treatment, which it licensed and now calls TAK-007, into pivotal studies in two types of lymphoma and CLL by 2021, with a potential filing for approval in 2023.

"Targeting CD19 was a proof of concept and now that we've demonstrated that this CAR NK approach can work and is safe we want to use this platform to target other types of cancers," said Rezvani, indicating interest in multiple myeloma and acute myeloid leukemia.

See the article here:
In small study, hints of promise for 'natural killer' cell therapy - BioPharma Dive

Theres no reason to be scared of genetically-modified marijuana. It could be the best bud youve ever had.

In 1996, the world was shocked when the news was announced that scientists in Scotland had cloned an animal. Dolly, as she was named, was an exact genetic duplicate of a female sheep.

To some extent, the researchers behind Dolly managed to achieve what no scientists before them had ever accomplished: they demonstrated humankinds mastery over its own genes.

What Dollys birth really represented, however, was humankinds mastery over our DNA, which is what makes up our genes. DNA is the instruction manual for how our biological processes operate. By birthing Dolly, we showed we can control genetic destiny.

One of the ways we are controlling our futures is by applying the techniques that created Dolly to change the genetic configurations of other living things, including plants.

This is the essence of genetic modification technology, and has led to the creation of genetically modified organisms, or GMOs.

GMO can be a bit of a dirty word, especially if youre a devotee of organic food. Many people think GMOs are unhealthy. But, where cannabis is concerned, genetic modification can allow us to improve how marijuana is produced.

The cannabis plant is a wonder of evolution, but in terms of maximizing the amount of medicinally useful molecules like THC and CBD that the plant produces, nature could use some juicing up.

As it stands, the only parts of the cannabis plant that produce psychoactive compounds are the trichomes small hair-like outgrowths on the surface of female cannabis plants flowers.

This means that the vast majority of the plants biomass, around 90%, from leaves to stalk to stems are useless from a commercial perspective.

So, imagine the possibilities if the cannabis plant could somehow be re-engineered so that it could produce those THC-bearing trichomes from root to tip. It would mean more than 90% of the plant could be used commercially, rather than being sent straight to the compost heap.

A trichome-saturated cannabis plant would also give producers much more bang for their buck, as it could produce more THC and/or CBD without more water, fertilizer or energy.

But the most radical proposal for engineering cannabis is to get rid of the plant altogether. Stay with me here it ends up making a lot of sense.

Because DNA is the universal genetic language that governs cells basic functions such as the manufacture of proteins, the replication of genetic material and the production of cellular energy they work the same way in many different organisms.

THC is the end result of a metabolic pathway. This means that a chemical, or combination of chemicals, is acted upon by an enzyme, or series of enzymes.

The starting chemicals are transformed by the enzyme into another chemical. In the case of cannabis, two chemicals in the plant, called geranyl pyrophosphate and olivetol, are combined by an enzyme called THCA synthase to form THC. The THC is then stored in the trichomes on the surface of the plant.

Now, the magic begins.

THCA synthase the enzyme that synthesizes THC in the cannabis plant is made out of protein. Because of this, we can determine the genetic sequence that encodes this protein.

This is easy to do, because the entire genetic sequence of the cannabis plant the cannabis genome has already been decoded, and this entire set of information is publically available.

Once you determine the genetic sequence that encodes the THCA synthase enzyme, you can use chemicals called restriction enzymes to clip that exact piece of DNA out of the strands of DNA that compose the cannabis genome.

Now, the researcher has a piece of DNA that encodes the THCA synthase enzyme, and nothing else. This chunk of genetic material can then be inserted into whatever type of organism you desire.

When you think of yeast, generally two things come to mind: beer and baking. But yeast represents a lot more than a glass of suds or a fresh-baked loaf of bread.

Yeast is actually a very powerful genetic organism. Yeast cells grow quickly in a culture solution at room temperature they will double in number every 100 minutes.

Now comes the fun part. Using some chemical tricks, we can take the segment of DNA we just snipped out of cannabis plant remember, the one that encodes the THCA synthase enzyme and pop it right into a yeast cells DNA.

This means that, as that single yeast cell copies itself and grows exponentially, from one to a sea of billions, it will ceaselessly manufacture the THCA enzyme as if that plant gene were a part of the very fabric of the yeast cell, which in fact it is.

So, if you put the THCA enzyme in a yeast cell, then grow that yeast cell in huge vats, youll eventually end up with a lot of THCA enzyme floating around in your vat.

The next step is to extract the enzyme from the solution, filtering out the cellular debris and growth medium. Once this is done, all you really need to do is add geranyl pyrophosphate and olivetolic to your THCA enzyme and voila, you have fully formed THC.

This THC can then be extracted from the growth medium; quality control-checked; packaged and sold at a hefty markup to a ravenous consumer market.

If using yeast cells to churn out cannabis products seems far-fetched, you might be surprised to know its actually happening now.

Hyasynth Bio, a company in Montreal, Canada, has developed their own strain of yeast cell that is genetically engineered to churn out cannabinoids.

The yeast cells are grown in giant vessels called bioreactors, each containing thousands of litres of growth medium, which are continually checked to ensure an absence of contamination and a proper growth environment.

Then, the yeast cells are disassembled; the yeast particles filtered out and the cannabinoids extracted from that solution.

Theres also another approach, one that may not sit well with purists. It involves making Frankenplants. But the future of genetically altered plants need not stoke horrific associations.

If anything, GMO weed could be the plant of the future, for a number of reasons.

GMO technology has gotten a very bad rap over the past couple of decades. Initial pushes to roll out GMO food crop staples things like tomatoes, wheat and corn that were engineered to be pest resistant were met with a huge amount of opposition, organized by environmental groups who had little knowledge of what they were protesting.

The world was shocked in the late 1990s and early 2000s when images flashed across TV screens around the world of protestors ripping up genetically-modified crops in test fields dressed in biohazard suits.

Many of these protests took place in the UK, which remains a hotbed of anti-GMO sentiment, led by organizations like Greenpeace UK, which is committed to boycotting genetic plant technologies.

As GMO crops began to be rolled out across North America, European activists tried to convince people on the other side of the pond that crops that had been genetically engineered were Frankenfoods and were to be avoided at all costs. They were met with some success.

But North Americans generally proved to be more tolerant to the idea that plants could be genetically modified and also safe to consume. That seems to be why countries in the western hemisphere seem to be leading the charge when it comes to GMO plant technologies, including cannabis.

There are several ways genetic modification technologies could enhance the experience of consuming your favourite strain of bud.

First, the cannabis plant could be engineered such that a tissue-specific promoter is added in front of the gene that produces the THCA enzyme.

A promoter is a small segment of DNA that is associated with a particular gene. It contains instructions that activate the gene it controls.

The promoter attached to the THCA gene would tell the gene to turn on in every part of the plant, such as its leaves, stem, etc. This means, in turn, the THCA enzyme would thus be active in every part of the cannabis plant, not just the buds.

Thus, every part of the plant including the parts youd never think of consuming would literally be dripping in THC, which could increase yields up to 90% for growers and producers of industrial cannabis.

With genetic modification technology, it seems the questionable environmental cost of large-scale cannabis production can be effectively mitigated.

The cannabis plant could also be engineered to require less water, or to express more photosynthetic pigments so it requires less energy to grow relative to a plant found in the wild.

But GMO technology doesnt just have the capability to dramatically increase yields of THC, CBD and other valuable compounds.

Because most plants grow based on DNA templates that are more or less interchangeable, traits from virtually any number of plant species could be spliced into the cannabis genome.

For example: imagine a Blueberry Kush strain that contains actual blueberry flavour compounds from the blueberry plant? Or a Unicorn sativa: a plant festooned with candy-coloured, multi-hued swirls of buds containing pigments from across the plant kingdom?

The future of cannabis is waiting to be engineered.

One of the ways this engineering is soon to happen will involve the CRISPR/Cas9 system. This genetic technology has gotten a lot of press recently, and for good reason.

CRISPR/Cas9 allows researchers to edit genomes from scratch with an incredible level of precision, which means certain traits can be custom-engineered.

In the most dystopian case, this means editing a human egg cell so the baby is sure to have blue eyes.

In a less ominous context, CRISPR/Cas9 could allow researchers to tweak the cannabis genome to get the plant to produce substances we would never be able to imagine, like fish proteins to protect against frost bite and citrus molecules that smell like a spring breeze

Lest that sound like science fiction, its already happening: Canopy Growth Corporation, one of the worlds largest legal cannabis companies, paid US$300 million to acquire Colorado-based Ebbu, a company that specializes in fiddling with cannabis genes using the CRISPR/Cas9 system.

As the technology becomes more and more widespread outside of niche applications in the biological sciences, expect more companies to crop up that use CRISPR/Cas9 to drive product development in the cannabis space.

No matter the technology used, or no matter what you think of GMOs, genetic manipulation of the cannabis plant is set to be a huge area of interest now and into the future. The tools of todays biological scientists make those used by Dollys engineers look positively paleolithic.

The best advice for thinking about the future of GMO weed: expect the unexpected.

See more here:
GMO Weed: The Future of Bud - Greencamp

Gene therapy can be defined as the treatment of a disease by modifying, replacing, or supplementing a genetic combination that is absent or abnormal and is responsible for causing causing the disease. Gene therapy has emerged as one of the most sought after research objectives in order to cure incurable diseases. Globally increasing instances of HIV, Cancer and other difficult to cure diseases is driving the demand for gene therapy.

This press release was orginally distributed by SBWire

New York, NY -- (SBWIRE) -- 02/10/2020 -- The global gene therapy market was valued at $393.35 million in 2018, and is estimated to reach $6,205.85 million by 2026, registering a CAGR of 34.8% from 2019 to 2026. Gene therapy is a technique that involves the delivery of nucleic acid polymers into a patient's cells as a drug to treat diseases. It fixes a genetic problem at its source. The process involves modifying the protein either to change the genetic expression or to correct a mutation. The emergence of this technology meets the rise in needs for better diagnostics and targeted therapy tools. For instance, genetic engineering can be used to modify physical appearance, metabolism, physical capabilities, and mental abilities such as memory and intelligence. In addition, it is also used for infertility treatment. Gene therapy offers a ray of hope for patients, who either have no treatment options or show no benefits with drugs currently available. The ongoing success has strongly supported upcoming researches and has carved ways for enhancement of gene therapy.

Major Key Players of the Gene Therapy Market are:Adaptimmune Therapeutics Plc., Anchiano Therapeutics Ltd., Achieve Life Sciences, Inc., Adverum Biotechnologies, Inc., Abeona Therapeutics Inc., Applied Genetic Technologies Corporation, Arbutus Biopharma Corporation, Audentes Therapeutics, Inc., AveXis, Inc., Bluebird Bio, Inc., Celgene Corporation, CRISPR Therapeutics AG, Editas Medicine, Inc., Editas Medicine, Inc., GlaxoSmithKline Plc., Intellia Therapeutics, Inc., Merck & Co., Inc., Novartis AG, REGENXBIO Inc., Spark Therapeutics, Inc., Sangamo Therapeutics, Inc., Uniqure N. V.,Voyager Therapeutics, Inc ,Amgen, Epeius Biotechnologies, Sanofi, Juno Therapeutics, Advantagene

Get sample copy of "Gene Therapy Market" at: https://www.marketgrowthinsight.com/sample/72026

This report on gene therapy covers different type of gene therapy developments, applications of gene therapy in curing diseases and market size in various geographical regions.-The report covers also cover gene therapy market developments from the development phase perspective as follows: Phase I, Phase II, Phase III-On the basis of applications of gene therapy, this report also covers all the major applications of gene therapy in curing major diseases, some of the major diseases covered in this report are as follows: Oncology, Infectious Diseases, Genetic Disorders, Cardiovascular Disorders, Diabetes Mellitus, Neurological Disorders and Others-This report has been further segmented into major regions, which includes detailed analysis of each region such as North America, Europe, Asia-Pacific (APAC) and Rest of the World (RoW) covering all the major country level markets for gene therapy in each of the region.

By Vector Type:

- Viral vectoro Retroviruseso Lentiviruseso Adenoviruseso Adeno Associated Viruso Herpes Simplex Viruso Poxviruso Vaccinia Viruso Others

- Non-viral vectoro Naked/Plasmid Vectorso Gene Guno Electroporationo Lipofectiono Others

By Gene Type- Antigen- Cytokine- Tumor Suppressor- Suicide- Deficiency- Growth factors- Receptors- Others

By Application- Oncological Disorders- Rare Diseases- Cardiovascular Diseases- Neurological Disorders- Infectious disease- Other Diseases

North America is the largest regional market for gene therapiesThe global market is segmented into four major regions, namely, North America, Europe, the Asia Pacific, and the Rest of the World. In 2018, North America accounted for the largest share of the market, followed by Europe. The rising prevalence of chronic diseases, high and growing healthcare expenditure, presence of advanced healthcare infrastructure, availability of reimbursements, and the presence of major market players in the region are the major factors driving the growth of the gene therapy market in North America.

Gene therapy involves modification of the faulty and missing gene/s and then delivery to the intended target using modified viral particles or other biotechnologically approved methods. This therapy is mostly considered as a one-time treatment; however, in some cases, it requires more than one dose of medication to completely cure the disease. Gene therapy, once considered impossible on a commercial-scale has now become a trend and most of the companies are banking on breakthrough innovations in the field. Many of the smaller companies have been successful in bringing few molecules to the market with the backing of larger companies.

Research objectives:- To study and analyze the global Gene Therapy consumption (value & volume) by key regions/countries, product type and application, history data. To understand the structure of the Gene Therapy Market by identifying its various sub-segments. Focuses on the key global Gene Therapy manufacturers, to define, describe and analyze the sales volume, value, market share, market competitive landscape, SWOT analysis, and development plans in the next few years. To analyze the Gene Therapy with respect to individual growth trends, future prospects, and their contribution to the total market. To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).

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Table of Content :

8.1. Adaptimmune Therapeutics Plc.8.1.1. Company Overview8.1.2. Company Snapshot8.1.3. Operating Business Segments8.1.4. Product Portfolio8.1.5. Business Performance8.1.6. Key Strategic Moves And Developments

8.2. Anchiano Therapeutics Ltd.8.2.1. Company Overview8.2.2. Company Snapshot8.2.3. Operating Business Segments8.2.4. Product Portfolio

8.3. Achieve Life Sciences, Inc.8.3.1. Company Overview8.3.2. Company Snapshot8.3.3. Operating Business Segments8.3.4. Product Portfolio8.3.5. Key Strategic Moves And Developments

8.4. Adverum Biotechnologies, Inc.8.4.1. Company Overview8.4.2. Company Snapshot8.4.3. Operating Business Segments8.4.4. Product Portfolio8.4.5. Key Strategic Moves And Developments

8.5. Abeona Therapeutics Inc.8.5.1. Company Overview8.5.2. Company Snapshot8.5.3. Operating Business Segments8.5.4. Product Portfolio8.5.5. Business Performance8.5.6. Key Strategic Moves And Developments

8.6. Applied Genetic Technologies Corporation8.6.1. Company Overview8.6.2. Company Snapshot8.6.3. Operating Business Segments8.6.4. Product Portfolio8.6.5. Business Performance8.6.6. Key Strategic Moves And Developments

8.7. Arbutus Biopharma Corporation8.7.1. Company Overview8.7.2. Company Snapshot8.7.3. Product Portfolio8.7.4. Business Performance8.7.5. Key Strategic Moves And Developments

8.8. Audentes Therapeutics Inc.8.8.1. Company Overview8.8.2. Company Snapshot8.8.3. Product Portfolio8.8.4. Key Strategic Moves And Developments

8.9. Avexis Inc.8.9.1. Company Overview8.9.2. Company Snapshot8.9.3. Product Portfolio8.9.4. Key Strategic Moves And Developments

8.10. Bluebird Bio, Inc.8.10.1. Company Overview8.10.2. Company Snapshot8.10.3. Operating Business Segments8.10.4. Product Portfolio8.10.5. Business Performance8.10.6. Key Strategic Moves And Developments

Have any query? Inquiry about report at: https://www.marketgrowthinsight.com/inquiry/72026

Many other therapies are also under development, which when approved and marketed, may contribute to significant revenue generation and would boost the industry growth. For instance, in 2018, there were 950 molecules in the development that were expected to be as effective treatment options for different indications like cancer, cardiac diseases, inherited blindness, and various other gene related defects.

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Gene Therapy Market Worldwide Industry Analysis and New Market Opportunities Explored, Forecast to 2026: Voyager Therapeutics, Sanofi, Juno...

Are you looking for some awesome memes about time travel? Then you have come to the right place.

Here we have hand-picked some of the best memes about time travel available on the internet. Some of them will also make you think!

Enjoy.

RELATED: THE ONE QUESTION EVERYONE WONDERS: IS TIME TRAVEL POSSIBLE?

It is a common theme in many a science fiction film and novel, but is time travel possible? As it turns out, it might just be -- but would be incrediblydifficult to achieve.

"Mathematically, you can certainly say something istravelingto the past," Liu said. But it is notpossiblefor you and me totravelbackward intime, he said. "However, some scientists believe thattravelingto the past is, in fact, theoreticallypossible, though impractical." -Live Science.

But, it should be notedthat traveling "forward" in time might actuallybe more plausible.

"Travellingforwardsin time is surprisinglyeasy. Einsteins special theory ofrelativity, developed in 1905, shows that time passes at different rates for people who are moving relative to one another - although the effect only becomes large when you get close to the speed of light." - physics.org.

Going back in time is, however, probably impossible. According to the principles of relativity, this would probably require you to travel faster than the speed of light.

This is a feat widely believed to be impossible. Some scientists have postulated that things called "wormholes" could be a way around this.

But they are not foolproof either. You could only travel back as far in time as when the wormhole itself was created.

"However, it would still be impossible to go back further in time than the point at which the wormhole was created, limiting the options for travel somewhat - and possibly explaining why we havent encountered any visitors from the future. If any natural wormholes were formed in the Big Bang, it might be possible to travel to a limited number of points in the past and in the distant universe, but wouldnt enable one to flit around the cosmos at will as the Doctor seems to do."- physics.org.

But, it should be notedthat there are some who believe that "time" itself is something ofan illusion.

"According to theoretical physicist Carlo Rovelli,timeis anillusion: our naive perception of its flow doesn't correspond to physical reality. Indeed, as Rovelli argues in The Order ofTime, much more is illusory, including Isaac Newton's picture of a universally ticking clock," - nature.com.

According to sites like popularmechanics.com, here are some of the best films about time travel: -

So, without further ado, here are some great memes about time travel. Trust us when we say this list is far from exhaustive and is in no particular order.

Here's an interesting ethical dilemma. If you could, would you go back in time to kill Hitler as a baby?

You would? But haven't you just killed an innocent baby?

Doesn't such an act make you as bad as people like Hitler?

Most believe that children are not born "evil" and are, in fact, products of their life's experiences, indoctrination into particular ideologies and upbringing, etc. Though some scientists have found links between brain physiology and anti-social traits like psychopathy.

So would killing baby Hitler be a "good thing?" You are, after all,judging an infant to death over a "precrime," like in theMinority Report.

Perhaps it would be better to have him raised by better parents?

But it might not matteranyway. If time travel was possible,people in the futurewould never have actuallyhave heard of him.

It would probably be best to keep it to yourself -- you have just killed a babyafter all.

Whenever time-travel is the main theme of a film, there is an unwritten rule that it must contain a certain amount of things. From unnecessarily complex scientific terminology to cliched quotes like "that's not how time travel works,"you might want toplay time-travel bingo next time you watch one.

It usually also has to include some ingenious, if slightly unhinged, scientist. This is simplya must.

A prime example is the recent Marvel blockbuster "Avengers: End Game."Thankfully, this film appears to have learned lessons from previous time-travel films and makes light of these many themes.

Quite refreshing.

Could this be proof that time travel is actuallypossiblein the future?"Dabbing" is a recent cultural phenomenon, isn't it?

Of course, in this photoshop day and age, we might not be able totrust the source of this image completely. Such sad times we live in.

It would be great to believe this image is, indeed, real, however.

But, in reality, this is actually a set pic from Christopher Nolan's recent blockbuster "Dunkirk."

We knew it. Everyone knows that the only reason Elon Musk is pushing mankind to colonize Mars is to fulfill his dreams of power.

And yet, there are still those who believe Musk's ambitions are purely altruistic. How could we have been so trusting?

Let's just hope he is a fair and magnanimous leader for the people of Mars.

With all the latest developments in genetic engineering, it can only be a matter of time before we get a real Jurassic Park? What could possibly go wrong?

But, given the warnings from Michael Crichton, perhaps it might not be a good idea.

After all, as Ian Malcolm famously said: "our scientists were so preoccupied with whether or not they could, they didnt stop to think if they should." Never a truer word was spoken.

But, that being said, it would be awesome!

Well, well, what have we here? With all those DeLoreans and a Tardis, this can only mean one thing -- A Time Traveller meetingof some kind.

This is one party we would certainlylove to be invited to. Oh, to be a fly on the wall.

But would they actuallyhave an "annual" meeting? It seems a little redundant.

And lastly, but by no means least, is this great quote from Carl Sagan. As it turns out, from a certain point of view, we have been able to travel to the past for as long as the written word has existed.

It is incredible to think that by reading ancient writers, like Marcus Aurelius,Aristotle, John Locke, or Thomas Aquinas (to name but a few), we can, in a way, talk to people from the past. This really is a mind-blowing thought when you think about it.

Now get out there and "stand on the shoulders of giants."

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7 Hilarious and Thought-Provoking Time Travel Memes - Interesting Engineering

CAMBRIDGE, Mass.(GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, is presenting new data from two of its development programs at Keystone Symposias Engineering the Genome Conference, a joint meeting with the Emerging Cellular Therapies: Cancer and Beyond Conference, taking place Feb. 8-12, 2020, in Banff, Canada. Intellia researchers are presenting data in support of the companys lead engineered cell therapy development candidate, NTLA-5001 for the treatment of the hematological cancer, acute myeloid leukemia (AML). Intellia also is sharing preclinical results for its hereditary angioedema (HAE) program, which is Intellias third CRISPR/Cas9 development program, announced in January 2020.

Intellia continues to demonstrate strong progress across both our engineered cell therapy and in vivo pipelines, said Intellia President and Chief Executive Officer John Leonard, M.D. We are observing very favorable preclinical data with our engineered T cells, and we are moving ahead with IND-enabling studies and manufacturing for NTLA-5001, to enable a regulatory submission in the first half of 2021.

On the in vivo side, the data from our HAE development program reinforce the modularity of Intellia's non-viral delivery genome editing platform and how it is enabling independent, single-dose therapies for multiple monogenic diseases. For HAE, we expect to nominate a development candidate in the first half of this year, continued Dr. Leonard.

New Data from Intellias Engineered Cell Therapy Development Program for AML

NTLA-5001, which is Intellias first engineered T cell therapy development candidate and is wholly owned, utilizes a T cell receptor (TCR)-directed approach to target the Wilms Tumor 1 (WT1) intracellular antigen for the treatment of AML. The companys WT1-TCR T cell approach aims to develop a broadly applicable treatment for AML patients, regardless of mutational background of a patients leukemia.

The company is presenting data demonstrating that the selection of a natural, high-affinity TCR, in combination with CRISPR-enabled engineering and targeted insertion, results in an engineered T cell capable of specific and potent killing of primary AML blasts. Todays presentation at Keystone builds on data previously presented last fall at the Annual Congress of the European Society of Gene and Cell Therapy (ESGCT).

The data being presented at the Keystone conference substantiate the advantages that a homogeneous T cell product developed through CRISPR engineering, like NTLA-5001, may have over traditional T cell engineering approaches. In particular, traditional T cell engineering methods typically result in a T cell product that carries two different TCRs, one endogenous and one transferred, which can pair in various combinations of alpha and beta chains and form mixed TCRs with unknown specificities. Intellia researchers are sharing today that the precise replacement of the endogenous TCR with the transgenic TCR (tgTCR) resulted in T cells with improved tgTCR expression levels and in 95% of edited T cells carrying exclusively the desired pairs of the tgTCR alpha and beta chains. This therapeutic TCR profile is expected to yield improved T cell product homogeneity, as researchers showed that Intellias T cell editing approach results in superior function of the engineered T cells toward WT1-positive targets in vitro. This therapeutic TCR profile is also expected to result in lower reactivity against unwanted targets on normal tissues that could lead to toxicities, including graft-versus-host disease (GvHD).

Researchers identified that the selected lead WT1 TCR exhibits high avidity (in the nM range) to its target epitope and shows tight epitope specificity. Being a natural TCR isolated from a healthy donor, it may have a lower cross-reactivity risk than many affinity-matured TCRs. Cells engineered with Intellia's lead WT1 TCR also demonstrated no detectable cytotoxicity toward bone marrow CD34+ cells, which express WT1 at low levels. This is an advantage over current CAR-T cell approaches targeting CD33 or CD123 in AML, which have been shown to induce severe bone marrow toxicity.

Furthermore, the data demonstrate that specific and potent killing of WT1-positive primary AML blasts result from T cells expressing Intellias lead WT1 TCR when cocultured in vitro. This outcome was observed across multiple patient samples that harbor the frequent HLA-A*02:01 allele and that express different WT1 levels as well as AML characteristics. These data validate that the epitope targeted by the lead WT1 TCR, which is distinct from a previously evaluated RMF epitope, is presented efficiently and broadly by AML tumor cells that carry the correct human leukocyte antigen (HLA) restriction. Intellias lead WT1 TCR also has the potential to target WT1-positive solid tumors, such as ovarian cancer, glioblastoma, lung cancer and mesothelioma.

The company plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the first half of 2021 for NTLA-5001 for the treatment of AML. Details on todays presentations on WT1 TCR T cells, including data from ongoing collaborations with researchers at IRCCS Ospedale San Raffaele, Milan, at Keystone are as follows:

First Data Presented on Potential CRISPR/Cas9-Based Therapy for HAE, Intellias Third Development Program

Researchers presented yesterday at the Keystone conference the companys first dataset in support of Intellias development program for HAE. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. The disease is caused by increased levels of the bradykinin protein. Most patients with HAE have a C1 esterase inhibitor (C1-INH) protein deficiency, which normally prevents the unregulated release and buildup of bradykinin.

Intellias HAE treatment hypothesis involves knocking out the kallikrein B1 (KLKB1) gene to reduce kallikrein activity, which is involved in the biological pathway for release of bradykinin. Intellia expects this reduction to correlate with a decrease in bradykinin activity, thus, preventing the activation of endothelial cells that causes vascular leakage and angioedema in HAE patients. The data presented at the Keystone conference showed that the knockout of KLKB1 produces in non-human primates (NHPs) a 90% reduction in kallikrein activity, a level that translates to a therapeutically meaningful impact on HAE attack rates (Source: Banerji et al., NEJM, 2017). This kallikrein activity reduction was sustained for at least five months in an ongoing NHP study, in a highly reproducible manner observed across both rodent and NHP studies.

Similar to its lead in vivo program, for the treatment of transthyretin amyloidosis (ATTR), Intellias potential HAE therapy utilizes the companys modular non-viral lipid nanoparticle (LNP) system to deliver CRISPR/Cas9. Intellias proprietary LNP-based delivery system includes two basic components: Cas9 messenger RNA (mRNA) and a guide RNA (gRNA). The gRNA is the only variable portion of the LNP delivery system and is the sole component that needs to be changed from the LNP-based delivery system that forms the foundation of NTLA-2001, Intellias development candidate for the treatment of ATTR for which the company intends to submit an IND application in mid-2020.

Intellia continues to evaluate several potential guide RNAs and expects to nominate a development candidate for HAE in the first half of 2020. Intellias KLKB1 HAE program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party.

Yesterdays short talk, titled In Vivo Delivery of CRISPR/Cas9 to the Liver Using Lipid Nanoparticles Enables Gene Knockout Across Multiple Targets in Rodent and Non-Human Primates, was made by Jessica Seitzer, director, genomics, Intellia. These data included results from ongoing collaborations with researchers at Regeneron.

All of Intellias presentations can be found here, on the Scientific Publications & Presentations page of Intellias website.

About Intellia Therapeutics

Intellia Therapeuticsis a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more aboutIntellia Therapeuticsand CRISPR/Cas9 atintelliatx.com and follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements ofIntellia Therapeutics, Inc.(Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an investigational new drug (IND) application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR) in mid-2020; its plans to submit an IND application for NTLA-5001, its first T cell receptor (TCR)-directed engineered cell therapy development candidate for its acute myeloid leukemia (AML) program in the first half of 2021; its plans to nominate a development candidate for its hereditary angioedema (HAE) program in the first half of 2020; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program, HAE program and other in vivo and ex vivo programs; its presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR, AML and HAE programs, in any future studies, including human clinical trials; its ability to develop other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; its business plans and objectives for its preclinical studies and clinical trials, including the therapeutic potential and clinical benefits thereof, as well as the potential patient populations that may be addressed by its ATTR program, AML program, HAE program and other in vivo and ex vivo programs; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc.(Regeneron) and Regenerons ability to enter into a Co/Co agreement for the HAE program; statements regarding the timing of regulatory filings for its development programs; its use of capital, including expenses, future accumulated deficit and other financial results during 2019 or in the future; and the ability to fund operations through the end of 2021.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain our intellectual property position; risks related to Intellias relationship with third parties, including our licensors; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; and the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with theSecurities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Jennifer Mound SmoterSenior Vice PresidentExternal Affairs & Communications+1 857-706-1071jenn.smoter@intelliatx.com

Lynnea OlivarezDirectorExternal Affairs & Communications+1 956-330-1917lynnea.olivarez@intelliatx.com

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

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Intellia Therapeutics Presents New Data From Its Engineered Cell Therapy and In Vivo Programs at Keystone Symposia's Engineering the Genome Conference...

ARTS WORK IN THE AGE OF BIOTECHNOLOGY: SHAPING OUR GENETIC FUTURES

Through Sunday, March 15

The Gregg Museum of Art & Design, Raleigh

Where do we draw the lines dividing art from science, natural from unnatural, and boldness from hubris?

An exhibit at N.C. States Gregg Museum of Art & Design doesnt answer these questions. Instead, it offers head-spinning new ways to ask them at the nexus of art and biotechnology, sharpening our insight into the fields future and expanding our understanding of it into the past.

These hard-to-classify collaborations between artists and scientistsseethe with hot-button issues related to ethics, privacy, human nature, and more. But if they have one message in common, its to be careful what you wish for.

Arts Work in the Age of Biotechnology: Shaping Our Genetic Futures is the result of more than two years of planning led by Molly Renda, the exhibit program librarian at N.C. State University Libraries, and the universitys Genetic Engineering and Society Center. Guest-curated by Hannah Star Rogers, who studies the intersection of art and science, the main exhibit at the Gregg has annexes in Hill and Hunt libraries.

On a recent tour of the exhibit, Renda and Fred Gould, the co-director of the GESC, said that they wanted to bring artists into the welter of science-and-design innovation taking place at the university because their differing perspectives on fundamental human issues create balance, tension, and discovery.

In the course of this, Ive found that artists tend to be more dystopian and designers are more utopian, Renda says.

There are different ways of knowing things, Gould adds. Thats why Molly came up with the name: not artwork, but arts work. What is an artist supposed to do?

Some pieces take on the dangers of day-after-tomorrow DNA testing and engineering technology. Heather Dewey-Hagborg is best known for Probably Chelsea, a piece in which she collected DNA samples from Chelsea Manning and generated thirty-two possible portraits of the soldier and activist.

When we worry about biotechnology, we usually worry that our food is going to be dangerous. But sometimes you wish for something thats rare: What happens when biotechnology makes it available to you?

The Gregg is showing a similar piece in which Dewey-Hagborg harvested DNA from cigarette butts and gum she found on the street and created probablebut not definitereplicas of the litterers faces, which hang on the walls above the specimens. Dewey-Hagborg demonstrates not only the unnerving extent of whats currently possible with DNA testing, but also the limits, which create misidentification risks.

Other pieces probe how biotechnology might reshape life as we know it. In a film and a sculpture representing an ancient Greek rite for women, Charlotte Jarvis raises the possibility of creating female sperm, based on the idea that, because stem cells are undifferentiated, you could theoretically teach womens stem cells to develop into sperm.

Still other pieces pointedly poke holes in the boundary between science and art. Adam Zaretskys Errorarium (entitled "Bipolar Flowers")looks like a cross between an arcade cabinet and a terrarium. It houses a few genetically modified Arabidopsis specimens, which Gould calls the white mice of research plants. When you turn the knobs, it changes the sonic parameters of a synthesizer, notionally testing the effects of the sound on the mutant plants.

It doesnt really do anythingor does it? Zaretskys experiment with no hypothesis is a playful tweak on science with something a little dangerous in the background.

Joe Davis, a bio-art pioneer, touches on something similar in his piece, which consists of documentation of an experiment where mice roll dice to determine if luck can be bred. Renda says that Davis couldnt get permission to run the test (universities are wary of drawing attention for ridiculous-seeming experiments), so he did it as conceptual art at N.C. State, instead.

Its notable that two artists home in on luck, one of many human concepts that genetic engineering, which will allow us to take control of our bodies and environment in untested ways, will transform. In We Make Our Own Luck Here, Ciara Redmond has bred four-leaf clovers (without genetic modification), which ruins themtheyrelucks evidence, not its cause. This whimsical iteration of unconsidered consequences raises a serious question: What else are we not thinking of?

When we worry about biotechnology, we usually worry that our food is going to be dangerous, Gould says. But sometimes you wish for something thats rare: What happens when biotechnology makes it available to you?

The exhibit takes an expansive view of biotechnology. Maria McKinney uses semen-extraction straws to sculpt proteins from double-muscled breeding bulls, underscoring that weve been tampering with life since long before CRISPR. Biotech feels radically new, but its revealed as part of a centuries-long process.

Another part of the exhibit, which closed at the end of October but can still be experienced through virtual reality at the Gregg, was From Teosinte to Tomorrow, Rendas land-art project at the North Carolina Museum of Art. In what was essentially a walk back through agricultural history, a bed of teosinte, which is thought to be the ancestor of modern maize, waited at the center of a corn maze.

That teosinte was in some sense genetically enhanced by subsistence farmers in Mexico since the time of the Aztecs, Gould says. Now were doing it in the laboratory with the same genesso whats the difference? Arts work is to make us think and question.

Contact arts and culture editor Brian Howe at bhowe@indyweek.com

Support independent local journalism.Join the INDY Press Clubto help us keep fearless watchdog reporting and essential arts and culture coverage viable in the Triangle.

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At the Crossroads of Art and Biotech, a Warning: Be Careful What You Wish For. - INDY Week

In his greenhouse at the Cold SpringHarbor Laboratory in Long Island, N.Y., plant geneticist Zach Lippman is growing cherry tomatoes.

But they don't look like the ones that most people grow in their gardens and greenhouses.

Lippman's tomatoes have shorter stemsand the fruit is more tightly clustered, looking more like grapes.

"With gene editing, we now have the ability to fine-tune at will," he said. "So instead of having black or white, small fruit [or] big fruit, you can have everything in between."

Lippman used CRISPR arevolutionarygene-editing tool that can quickly and precisely edit DNA to tweak three of the plant's genes, and make them suitable for large-scale urban agriculture for the first time.

With CRISPR, researchers can precisely target and cut any kind of genetic material. Don't want your mushrooms to turn brown after a few days? Remove the gene that causes thatand problem solved.

There's a lot of excitement about the introduction of gene-edited products into the Canadian food system over the next few years, but a lot of trepidation as well.

The food industry's last foray into genetic engineering genetically modified organisms (GMOs) in the 1990s was a financial success. But the practice is an ongoing public relations nightmare, as many Canadians remain wary of products critics have labelled "Frankenfoods."

Currently, the only gene-edited product commercially available is a soybean oil being used by a restaurant chain in the American Midwest for cooking and salad dressings. It has a longer shelf life than other cooking oils and produces less saturated fat and no trans fat.

Ian Affleck, vice-president of plant biotechnology at CropLife Canada, a trade association that represents Canadian manufacturers of pesticides and plant-breeding products, estimates the soybean oil might be in Canada in a year or two, followed by some altered fruits and vegetables.

Even then, he said, supplies will likely be limited while farmers and food companies determine if consumers will embrace genetically edited food.

All the major health organizations in the world, including Health Canada, have concluded that eating GMO foods does not pose eithershort or long-term health risks.

According to the World Health Organization, GMO goods currently approved for the market "have passed safety assessments and are not likely to present risks for human health."

But Canadians remain stubbornly unconvinced even though about 90 per cent of the corn, soybeansand canola grown in Canada is genetically modified, as is almost all of the processed food we consume.

A 2018 pollby market research company Statista found only 37 per cent of people surveyed strongly or somewhat strongly agreed that GMOs were safe to eat, while 34 per cent strongly or somewhat strongly disagreed.

Industry representatives now say they spent too much time marketing their GMOproducts to farmersand not enough time communicating the benefitsto consumers.

"We spoke to two per cent of the population, who are those who farm," said Affleck. "And those who opposed the technology spoke to the other 98 per cent of the population."

"We thought it was just another transition in plant breeding," recalled Stuart Smyth, who holds the University of Saskatchewan's industry-funded research chair in agri-food innovation. "Nobody expected the environmental groups to develop into a political opposition."

With gene-edited foods, Smyth believes the industry needs to focus on public education to counteract what he calls the "propaganda" that will be coming from the other side.

Gene-edited foods will differ from GMOs in one important respect.

When foods are genetically modified, foreign genes are often added to an existing genome. If you want a vegetable to grow better in cold weather, you could add a gene from a fish that lives in icy water.That's what earned GMO products the "Frankenfoods" moniker.

With gene-editing tools like CRISPR, genes can be cut out, or "turned off," but nothing new is added to the genome.

Lucy Sharratt, co-ordinator of the Canadian Biotechnology Action Network, isn't convinced there's a significant difference.

"The new techniques of gene editing are clearly techniques of genetic engineering," she said. "They are all invasive methods of changing a genome directly at the molecular level.

"While we can produce organisms with new traits, that doesn't mean we know exactly all of what we've done to that organism. There can be many unintended effects," Sharratt further argued.

Unlike GMOs, which require extensive regulatory approval before going to market, gene-edited foods will likely appear without undergoing a risk assessment by Canadian regulators.

Health Canada doesn't require safety testing for new products if it determines those products aren't introducing "novel traits" into the food system. Since it considers gene editing to be an extension of traditional plant breeding, no stamp of approval will be necessary.

That concerns Jennifer Kuzma, co-director of the Genetic Engineering and Society Center at North Carolina State University, whothinks gene-edited products should be tracked and monitored "for those low-level health effects that some products might be contributing to."

Sharratt is also skeptical that gene editing will produce the benefits its supporters claim, pointing to "a biotech industry that has oversold technology and made all kinds of broad promises for the use of genetic engineering that didn't come to pass." Things like reduced pesticide use and greater drought resistance, for example.

Kuzma agrees that GMO researchers have sometimes been guilty of "perhaps overstating the promise of the technology and understating potential risk."But she believes those involved in developing gene-editing techniques want to avoid repeating the mistakes of the past.

"They have a really sincere desire to be more open and transparent in the ways that they communicate and in the sharing of information," she said. "They do realize that the first generation of genetic engineering did not go so well from a public confidence perspective."

The GMO food industry has fiercely opposed one of the most obvious methods to boost public confidence: mandatory labelling, even as a 2018 survey from Dalhousie University showed an overwhelming majority of Canadians support it.

Sixty-four countries require mandatory labelling for GMO products. Canada is not one of them.

There are no plans to require mandatory labelling of gene-edited foods, either.

Jonathan Latham, executive director of the Bioscience Resource Project, a New York-based non-profit organization that researches genetic engineering, thinks that's a mistake.

"If you want people to make informed decisions and you want them to make that in a democratic fashion, then the more information you give them, the better," he said. "And so to deny people information about the content of their food is to violate a very basic democratic right."

Lathamalso believes that not labelling genetically engineered productsincreases consumer skepticism.

"[Consumers] don't really understand why, if a company wants to produce a product and advertise it and tell everybody how good it is, why they shouldn't also want to label it," he said.

Sharratt would like to see Canada adopt the approach taken by the European Court of Justice, which ruled in 2018 that gene-edited foods must undergo the same testing as GMOs before being allowed on grocery store shelves.

Lippman doesn't believe that will happen. In fact, he thinks the potential of gene-edited foods is so great that the public will demand even greater access to suchproducts.

"People will start to be educated and see that there's nothing harmful about it. It's completely fine. And then the only issue sticking out there will be whether we're over-promising.That'll be it."

Click 'listen' above to hear Ira Basen's documentary, The Splice of Life.

Read more here:
Gene editing could revolutionize the food industry, but it'll have to fight the PR war GMO foods lost - CBC.ca

Last year left us with this piece of bombshell news: He Jiankui, the mastermind behind the CRISPR babies scandal, has been sentenced to three years in prison for violating Chinese laws on scientific research and medical management. Two of his colleagues also face prison for genetically engineering human embryos that eventually became the worlds first CRISPRd babies.

The story isnt over: at least one other scientist is eagerly following Hes footsteps in creating gene-edited humans, although he stresses that he wont implant any engineered embryos until receiving regulatory approval.

Biotech stories are rarely this dramatic. But as gene editing tools and assisted reproductive technologies increase in safety and precision, were bound to see ever more mind-bending headlines. Add in a dose of deep learning for drug discovery and synthetic biology, and its fair to say were getting closer to reshaping biology from the ground upboth ourselves and other living creatures around us.

Here are two stories in biotech were keeping our eyes on. Although successes likely wont come to fruition this year (sorry), these futuristic projects may be closer to reality than you think.

The idea of human-animal chimeras immediately triggers ethical aversion, but the dream of engineering replacement human organs in other animals is gaining momentum.

There are two main ways to do this. The slightly less ethically-fraught idea is to grow a fleet of pigs with heavily CRISPRd organs to make them more human-like. It sounds crazy, but scientists have already successfully transplanted pig hearts into baboonsa stand-in for people with heart failurewith some recipients living up to 180 days before they were euthanized. Despite having foreign hearts, the baboons were healthy and acted like their normal buoyant selves post-op.

But for cross-species transplantation, or xenotransplants to work in humans, we need to deal with PERVsa group of nasty pig genes scattered across the porcine genome, remnants of ancient viral infections that can tag along and potentially infect unsuspecting human recipients.

Theres plenty of progress here too: back in 2017 scientists at eGenesis, a startup spun off from Dr. George Churchs lab, used CRISPR to make PERV-free pig cells that eventually became PERV-free piglets after cloning. Then last month, eGenesis reported the birth of Pig3.0, the worlds most CRISPRd animal to further increase organ compatibility. These PERV-free genetic wonders had three pig genes that stimulate immunorejection removed, and nine brand new human genes to make themin theorymore compatible with human physiology. When raised to adulthood, pig3.0 could reproduce and pass on their genetic edits.

Although only a first clinical propotype that needs further validation and refinement, eGenesis is hopeful. According to one (perhaps overzealous) estimate, the first pig-to-human xenotranplant clinical trial could come in just two years.

The more ethically-challenged idea is to grow human organs directly inside other animalsin other words, engineer human-animal hybrid embryos and bring them to term. This approach marries two ethically uncomfortable technologies, germline editing and hybrids, into one solution that has many wondering if these engineered animals may somehow receive a dose of humanness by accident during development. What if, for example, human donor cells end up migrating to the hybrid animals brain?

Nevertheless, this year scientists at the University of Tokyo are planning to grow human tissue in rodent and pig embryos and transplant those hybrids into surrogates for further development. For now, bringing the embryos to term is completely out of the question. But the line between humans and other animals will only be further blurred in 2020, and scientists have begun debating a new label, substantially human, for living organisms that are mainly human in characteristicsbut not completely so.

With over 800 gene therapy trials in the running and several in mature stages, well likely see a leap in new gene medicine approvals and growth in CAR-T spheres. For now, although transformative, the three approved gene therapies have had lackluster market results, spurring some to ponder whether companies may cut down on investment.

The research community, however, is going strong, with a curious bifurcating trend emerging. Let me explain.

Genetic medicine, a grab-bag term for treatments that directly change genes or their expression, is usually an off-the-shelf solution. Cell therapies, such as the blood cancer breakthrough CAR-T, are extremely personalized in that a patients own immune cells are genetically enhanced. But the true power of genetic medicine lies in its potential for hyper-personalization, especially when it comes to rare genetic disorders. In contrast, CAR-Ts broader success may eventually rely on its ability to become one-size-fits-all.

One example of hyper-tailored gene medicine success is the harrowing story of Mila, a six-year-old with Batten disease, a neurodegenerative genetic disorder that is always fatal and was previously untreatable. Thanks to remarkable efforts from multiple teams, however, in just over a year scientists developed a new experimental therapy tailored to her unique genetic mutation. Since receiving the drug, Milas condition improved significantly.

Milas case is a proof-of-concept of the power of N=1 genetic medicine. Its unclear whether other children also carry her particular mutationBatten has more than a dozen different variants, each stemming from different genetic miscodingor if anyone else would ever benefit from the treatment.

For now, monumental costs and other necessary resources make it impossible to pull off similar feats for a broader population. This is a shame, because inherited diseases rarely have a single genetic cause. But costs for genome mapping and DNA synthesis are rapidly declining. Were starting to better understand how mutations lead to varied disorders. And with multiple gene medicines, such as antisense oligonucleotides (ASOs) finally making a comeback after 40 years, its not hard to envision a new era of hyper-personalized genetic treatments, especially for rare diseases.

In contrast, the path forward for CAR-T is to strip its personalization. Both FDA-approved CAR-T therapies require doctors to collect a patients own immune T cells, preserved and shipped to a manufacturer, genetically engineered to boost their cancer-hunting abilities, and infused back into patients. Each cycle is a race against the cancer clock, requiring about three to four weeks to manufacture. Shipping and labor costs further drive up the treatments price tag to hundreds of thousands of dollars per treatment.

These considerable problems have pushed scientists to actively research off-the-shelf CAR-T therapies, which can be made from healthy donor cells in giant batches and cryopreserved. The main stumbling block is immunorejection: engineered cells from donors can cause life-threatening immune problems, or be completely eliminated by the cancer patients immune system and lose efficacy.

The good news? Promising results are coming soon. One idea is to use T cells from umbilical cord blood, which are less likely to generate an immune response. Another is to engineer T cells from induced pluripotent stem cells (iPSC)mature cells returned back to a young, stem-like state. A patients skin cells, for example, could be made into iPSCs that constantly renew themselves, and only pushed to develop into cancer-fighting T cells when needed.

Yet another idea is to use gene editing to delete proteins on T cells that can trigger an immune responsethe first clinical trials with this approach are already underway. With at least nine different off-the-shelf CAR-T in early human trials, well likely see movement in industrialized CAR-T this year.

Theres lots of other stories in biotech we here at Singularity Hub are watching. For example, the use of AI in drug discovery, after years of hype, may finally meet its reckoning. That is, can the technology actually speed up the arduous process of finding new drug targets or the design of new drugs?

Another potentially game-changing story is that of Biogens Alzheimers drug candidate, which reported contradicting results last year but was still submitted to the FDA. If approved, itll be the first drug to slow cognitive decline in a decade. And of course, theres always the potential for another mind-breaking technological leap (or stumble?) thats hard to predict.

In other words: we cant wait to bring you new stories from biotechs cutting edge in 2020.

Image Credit: Image by Konstantin Kolosov from Pixabay

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Poplar trees, which emit trace amounts of the gas isoprene, can be genetically modified not to harm air quality while leaving their growth potential unchanged, according to new research.

While providing benefits to the environment, some trees, like poplars, also emit gases to the atmosphere that worsen air pollution and alter climate.

The findings in the Proceedings of the National Academy of Sciences are important because poplar plantations cover 9.4 million hectares (36,294 square miles) globallymore than double the land used 15 years ago. Poplars are fast-growing trees that are a source of biofuel and other products including paper, pallets, plywood, and furniture frames.

Poplars and other trees used in plantation agroforestry, including palms and eucalyptus, produce isoprene in their leaves in response to climate stress such as high temperature and drought. The isoprene alleviates those stresses by signaling cellular processes to produce protective molecules; however, isoprene is so volatile that millions of metric tons leak into the atmosphere each year.

The isoprene reacts with gases that tailpipe pollution produces to create ozone, which is a respiratory irritant. Isoprene also causes higher levels of atmospheric aerosol production, which reduces the amount of direct sunlight reaching the earth (a cooling effect), and it causes the global warming potential of methane in the atmosphere to increase (a warming effect). The warming effect is most likely greater than the cooling effect. The net effect of emitted isoprene is to worsen respiratory health and, most likely, warm the atmosphere.

Researchers genetically modified poplars not to produce isoprene, then tested them in three- and four-year trials at plantations in Oregon and Arizona.

They found that trees whose isoprene production was genetically suppressed did not suffer ill effects in terms of photosynthesis or biomass production. They were able to make cellulose, used in biofuel production, and grow as well as trees that were producing isoprene. The discovery came as a surprise, given the protective role of isoprene in stressful climates, especially in the case of the Arizona plantation.

The suppression of isoprene production in the leaves has triggered alternative signaling pathways that appear to compensate for the loss of stress tolerance due to isoprene, says lead author Russell Monson, a professor of ecology and evolutionary biology at the University of Arizona. The trees exhibited a clever response that allowed them to work around the loss of isoprene and arrive at the same outcome, effectively tolerating high temperature and drought stress.

Our findings suggest that isoprene emissions can be diminished without affecting biomass production in temperate forest plantations, says coauthor Steven Strauss, a distinguished professor of forest biotechnology at Oregon State University.

Thats what we wanted to examinecan you turn down isoprene production, and does it matter to biomass productivity and general plant health? It looks like it doesnt impair either significantly.

To modify the poplars, the researchers used a genetic engineering tool known as RNA interference. RNA transmits protein coding instructions from each cells DNA, which holds the organisms genetic code. Scientists at the Institute of Biochemical Plant Pathology at the Helmholtz Research Center in Munich, Germany who collaborated on the study developed the genetic tools for modifying the trees, and the protein analyses that revealed changes in the use of biochemical pathways.

RNA interference is like a vaccinationit triggers a natural and highly specific mechanism whereby specific targets are suppressed, be they the RNA of viruses or endogenous genes, Strauss says.

You could also do the same thing through conventional breeding. It would be a lot less efficient and precise, and it might be a nightmare for a breeder who may need to reassess all of their germplasm and possibly exclude their most productive cultivars as a result, but it could be done. New technologies like CRISPR, short for clustered regularly interspaced short palindromic repeats, which allows for precise DNA editing at specific stretches of the genetic code, should work even better.

In an additional discovery, the researchers found that trees were able to adjust to the loss of isoprene because most plantation growth takes place during cooler and wetter times of the year.

This means that, for this species, the natural seasonal cycle of growth works in favor of high biomass production when the beneficial effects of isoprene are needed least, Monson explains.

This observation also clarified an adaptive role for isoprene in natural forests, where protection that enhances survival during mid-season climate stress is likely more important than processes that promote growth early in the season.

The fact that cultivars of poplar can be produced in a way that ameliorates atmospheric impacts without significantly reducing biomass production gives us a lot of optimism, Monson says.

Were striving toward greater environmental sustainability while developing plantation-scale biomass sources that can serve as fossil fuel alternatives.

Additional researchers from Portland State University; the University of California, Riverside; NASAs Goddard Space Flight Center; and the Institute for Microbiology in Greifswald, Germany also collaborated on the study.

Funding came, in part, from the National Science Foundation, the US Department of Agriculture, and the German Ministry of Education and Research.

Source: Russell Monson for University of Arizona

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Genetically modified poplar trees won't pollute the air - Futurity: Research News